Real-World Effectiveness and Safety of Mepolizumab in Pediatric Severe Eosinophilic Asthma: A Multicenter Study


Çevik N. N., OCAK M., Kocatepe G., Teker N. B., KOCACIK UYGUN D. F., SERBES M., ...Daha Fazla

Pediatric Pulmonology, cilt.61, sa.1, 2026 (SCI-Expanded, Scopus) identifier identifier

  • Yayın Türü: Makale / Tam Makale
  • Cilt numarası: 61 Sayı: 1
  • Basım Tarihi: 2026
  • Doi Numarası: 10.1002/ppul.71466
  • Dergi Adı: Pediatric Pulmonology
  • Derginin Tarandığı İndeksler: Science Citation Index Expanded (SCI-EXPANDED), Scopus, EMBASE, MEDLINE
  • Anahtar Kelimeler: Anti-IL-5, children, Mepolizumab, Severe eosinophilic asthma
  • Akdeniz Üniversitesi Adresli: Evet

Özet

Background: Severe asthma (SA) in children is a complex condition with high morbidity and healthcare costs. Mepolizumab, an anti-interleukin-5 biologic, is approved for severe eosinophilic asthma (SEA) in patients ≥ 6 years, yet long-term real-world pediatric data remain limited. Aim: To assess the long-term safety and effectiveness of mepolizumab in pediatric SEA, focusing on lung function, oral corticosteroid (OCS) use, and exacerbation rates. Methods: This retrospective, multicenter study examined the medical records of 33 patients with SEA (aged 6–17 years) who received mepolizumab treatment at three tertiary centers in Turkiye. Inclusion criteria included GINA-defined SA, ≥ 2 severe exacerbations in the previous year requiring OCS, high-dose ICS plus a second controller, and elevated eosinophil counts. Data on exacerbations, pulmonary function tests (PFTs), OCS use, ACT scores, eosinophils, and adverse events were collected over 24 months. Results: At baseline, patients showed poor asthma control (median ACT: 13), impaired lung function (FEV1%: 62%), and frequent exacerbations (median: 7/year). Mepolizumab significantly reduced exacerbation rates (median: 7 to 0–0.05 at 12/24 months, p = 0.005) and OCS use (87.9% OCS-free by 3 months). ACT scores improved (median: 13–25, p < 0.001), as did FEV1% (62% to 89%, p < 0.001) at 24 months. Eosinophil counts decreased markedly (460 to 50 cells/µL, p < 0.001). The treatment was well-tolerated; one patient discontinued due to anaphylaxis and four due to lack of efficacy. Conclusion: Mepolizumab showed sustained effectiveness and good tolerability in pediatric SEA, significantly reducing exacerbations and OCS use while improving asthma control and lung function. These findings support its real-world utility and underscore the need for careful patient selection and monitoring.